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DE60042984D1 - Drugs for the treatment of Duchenne muscular dystrophy - Google Patents

Drugs for the treatment of Duchenne muscular dystrophy

Info

Publication number
DE60042984D1
DE60042984D1 DE60042984T DE60042984T DE60042984D1 DE 60042984 D1 DE60042984 D1 DE 60042984D1 DE 60042984 T DE60042984 T DE 60042984T DE 60042984 T DE60042984 T DE 60042984T DE 60042984 D1 DE60042984 D1 DE 60042984D1
Authority
DE
Germany
Prior art keywords
drugs
treatment
muscular dystrophy
duchenne muscular
duchenne
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Lifetime
Application number
DE60042984T
Other languages
German (de)
Inventor
Masafumi Matsuo
Yasuhiro Takeshima
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
JCR Pharmaceuticals Co Ltd
Original Assignee
JCR Pharmaceuticals Co Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by JCR Pharmaceuticals Co Ltd filed Critical JCR Pharmaceuticals Co Ltd
Application granted granted Critical
Publication of DE60042984D1 publication Critical patent/DE60042984D1/en
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/06—Anabolic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
DE60042984T 2000-04-26 2000-09-27 Drugs for the treatment of Duchenne muscular dystrophy Expired - Lifetime DE60042984D1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP2000125448 2000-04-26

Publications (1)

Publication Number Publication Date
DE60042984D1 true DE60042984D1 (en) 2009-10-29

Family

ID=18635405

Family Applications (2)

Application Number Title Priority Date Filing Date
DE60042984T Expired - Lifetime DE60042984D1 (en) 2000-04-26 2000-09-27 Drugs for the treatment of Duchenne muscular dystrophy
DE60039038T Expired - Lifetime DE60039038D1 (en) 2000-04-26 2000-09-27 Splicing enhancer sequences in the dystrophy gene

Family Applications After (1)

Application Number Title Priority Date Filing Date
DE60039038T Expired - Lifetime DE60039038D1 (en) 2000-04-26 2000-09-27 Splicing enhancer sequences in the dystrophy gene

Country Status (6)

Country Link
US (1) US6653467B1 (en)
EP (2) EP1544297B1 (en)
JP (2) JP4590033B2 (en)
AU (1) AU780517B2 (en)
CA (1) CA2319149A1 (en)
DE (2) DE60042984D1 (en)

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US6727355B2 (en) * 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US7902160B2 (en) * 2002-11-25 2011-03-08 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP3808845A1 (en) 2004-06-28 2021-04-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
FR2874384B1 (en) * 2004-08-17 2010-07-30 Genethon ADENO-ASSOCIATED VIRAL VECTOR FOR PRODUCING EXON JUMP IN A GENE ENCODING A PROTEIN WITH DISPENSABLE DOMAINS
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
AU2006237727B2 (en) * 2005-04-22 2012-06-28 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mRNA by interfering with the binding of SR proteins and by interfering with secondary RNA structure.
US20070017138A1 (en) * 2005-04-25 2007-01-25 Young Kevin D Weapon grip assist
WO2007123391A1 (en) * 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US8097596B2 (en) * 2006-06-30 2012-01-17 Lakewood-Amedex, Inc. Compositions and methods for the treatment of muscle wasting
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
CA2942716C (en) * 2007-07-12 2019-07-09 Biomarin Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
EP2167135A2 (en) * 2007-07-12 2010-03-31 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
PT2203173E (en) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
EP2249874A1 (en) * 2008-02-08 2010-11-17 ProSensa Holding BV Methods and means for treating dna repeat instability associated genetic disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
BR122020021379B1 (en) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. morpholino phosphorodiamidate oligomer, composition comprising the same and use of said oligomer to treat muscular dystrophy
JP2012524540A (en) 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Oligonucleotides containing inosine for treating DMD
DK2499249T3 (en) 2009-11-12 2018-12-03 Univ Western Australia ANTISENCE MOLECULES AND PROCEDURES FOR TREATING PATHOLOGIES
JP6141018B2 (en) 2009-12-24 2017-06-07 バイオマリン テクノロジーズ ベー．フェー． Molecules for treating inflammatory disorders
CA2806616C (en) * 2010-08-20 2015-08-11 Replicor Inc. Oligonucleotide chelate complexes
TWI541024B (en) 2010-09-01 2016-07-11 日本新藥股份有限公司 Antisense nucleic acid
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
ES2633565T3 (en) 2010-11-12 2017-09-22 The General Hospital Corporation Non-coding RNAs associated with polycomb
EP2672977A1 (en) * 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
US20130085139A1 (en) * 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CN112251436A (en) 2012-01-27 2021-01-22 比奥马林技术公司 RNA-regulated oligonucleotides with improved properties for the treatment of duchenne muscular dystrophy and becker muscular dystrophy
EP3738974A1 (en) 2012-09-28 2020-11-18 The University of North Carolina at Chapel Hill Aav vectors targeted to oligodendrocytes
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
WO2014144978A2 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
US10858650B2 (en) 2014-10-30 2020-12-08 The General Hospital Corporation Methods for modulating ATRX-dependent gene repression
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
AU2016215124B2 (en) 2015-02-06 2020-08-06 The University Of North Carolina At Chapel Hill Optimized human clotting Factor VIII gene expression cassettes and their use
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
MA41795A (en) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc EXCLUSION OF AN EXON INDUCED BY ANTISENSE COMPOUNDS IN MYOSTATIN
JP2019525742A (en) 2016-06-30 2019-09-12 サレプタ セラピューティクス， インコーポレイテッド Exon skipping oligomer for muscular dystrophy
CN121271865A (en) * 2016-07-05 2026-01-06 比奥马林技术公司 Precursor mRNA splicing conversion or regulatory oligonucleotides containing a double-loop scaffold portion that have therapeutic effects on genetic diseases
HUE059843T2 (en) 2016-12-19 2023-01-28 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
CA3047010A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
PT4122497T (en) 2016-12-19 2024-06-12 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
EA201991450A1 (en) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. OLIGOMER CONJUGATES FOR EXONISM SKIP IN MUSCULAR DYSTROPHY
SG11202010841QA (en) * 2018-05-11 2020-11-27 Alpha Anomeric Sas Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP4659766A2 (en) 2018-06-12 2025-12-10 The University of North Carolina at Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
EP4146673A4 (en) 2020-05-05 2024-06-19 The University of North Carolina at Chapel Hill Modified adeno-associated virus 5 capsids and uses thereof

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JP2000325085A (en) * 1999-05-21 2000-11-28 Masafumi Matsuo Pharmaceutical composition for treatment of duchenne muscular dystrophy
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US6653467B1 (en) * 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
JP4836366B2 (en) * 2000-08-25 2011-12-14 雅文 松尾 Duchenne muscular dystrophy treatment
US6727355B2 (en) * 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy

Also Published As

Publication number Publication date
EP1544297A2 (en) 2005-06-22
EP1160318B1 (en) 2008-05-28
JP2010268815A (en) 2010-12-02
EP1544297B1 (en) 2009-09-16
JP2002010790A (en) 2002-01-15
US6653467B1 (en) 2003-11-25
EP1160318A2 (en) 2001-12-05
EP1544297A3 (en) 2005-07-06
AU780517B2 (en) 2005-03-24
CA2319149A1 (en) 2001-10-26
DE60039038D1 (en) 2008-07-10
EP1160318A3 (en) 2004-06-23
JP4590033B2 (en) 2010-12-01
AU6135400A (en) 2001-11-01

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Legal Events

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